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Repair Biotechnologies Receives Orphan Drug Designation From the FDA for the Treatment of Homozygous Familial Hypercholesterolemia

News Provided by ACCESS Newswire2025-05-19

SYRACUSE, NEW YORK / ACCESS Newswire / May 19, 2025 /Repair Biotechnologies, Inc. (https://www.repairbiotechnologies.com/) is a biotechnology company developing first-in-class therapies capable of rapidly reducing atherosclerotic plaque. The company is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its drug REP-0003 for the treatment of patients with homozygous familial hypercholesterolemia (HoFH), a rare condition of accelerated atherosclerosis.

"REP-0003 is a cutting-edge messenger RNA therapy that clears harmful excess free cholesterol inside cells while preserving essential cholesterol, a potential breakthrough for the treatment of severe atherosclerosis in HoFH patients," said Mourad Topors, CSO at Repair Biotechnologies. "REP-0003 has consistently produced sizable regression of atherosclerotic plaque, restored liver function, and improved exercise capacity in the LDLR-knockout mouse model of HoFH. As in humans with HoFH, these mice lack functional low-density lipoprotein receptors (LDLR) and consequently exhibit impaired cholesterol metabolism and dramatically accelerated growth of atherosclerotic plaque. We are excited by the transformative potential of REP-0003 and look forward to working with the FDA to bring this potentially life-changing treatment to patients."

REP-0003 was developed using Repair Biotechnologies ' proprietary Cholesterol Degrading Platform (CDP), and provides a way to safely break down and eliminate the undruggable target of toxic excess intracellular cholesterol in tissues. The company plans to begin a first clinical trial of REP-0003 in 2026.

"The Orphan Drug Designation is a major milestone for our program, and acknowledges the continued and significant unmet need in the HoFH community," said Reason, Repair Biotechnologies ' CEO. "It is an important step forward in our efforts to bring the first potentially curative therapy to the clinic for HoFH patients with severe, life-threatening atherosclerotic plaque."

The Orphan Drug Designation grants seven years of market exclusivity upon approval for the treatment of patients with HoFH. It also provides significant financial incentives, including tax credits for clinical trial costs and waived FDA user fees, easing the development burden. Additionally, the designation offers specialized FDA guidance and potential fast-track pathways, accelerating the path to market for life-changing therapies.

Contact Information

Reason
CEO and Co-Founder
press@repairbiotechnologies.com
+1 315-299-2407